UMIN-CTR Clinical Trial
| Unique ID issued by UMIN | UMIN000028429 |
|---|---|
| Receipt number | R000032495 |
| Scientific Title | Multicenter randomized double-blind comparison test followed by open-label continuous administration test of NPC-12T for Fibrodysplasia Ossificans Progressiva |
| Date of disclosure of the study information | 2017/08/01 |
| Last modified on | 2023/08/03 12:10:53 |
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Basic information
Public title
Multicenter randomized double-blind comparison test followed by open-label continuous administration test of NPC-12T for Fibrodysplasia Ossificans Progressiva
Acronym
Investigator-initiated clinical trial of NPC-12T for Fibrodysplasia Ossificans Progressiva
Scientific Title
Multicenter randomized double-blind comparison test followed by open-label continuous administration test of NPC-12T for Fibrodysplasia Ossificans Progressiva
Scientific Title:Acronym
Investigator-initiated clinical trial of NPC-12T for Fibrodysplasia Ossificans Progressiva
Region
| Japan |
Condition
Condition
Fibrodysplasia Ossificans Progressiva
Classification by specialty
| Pediatrics | Orthopedics | Rehabilitation medicine |
Classification by malignancy
Others
Genomic information
NO
Objectives
Narrative objectives1
To investigate the clinical efficacy and safety of NPC-12T for Fibrodysplasia Ossificans Progressiva by multicenter randomized double-blind placebo-controlled comparison test followed by open-label continuous administration test
Basic objectives2
Safety,Efficacy
Basic objectives -Others
Trial characteristics_1
Exploratory
Trial characteristics_2
Others
Developmental phase
Phase II,III
Assessment
Primary outcomes
Objective evaluation of physical function using J-HAQ (or J-CHAQ) at the end of double-blind stage
Key secondary outcomes
Frequency and grade of adverse events and side effects
Quantitative assessment of heterotopic bone by whole-body CT
Number of newly formed heterotopic bone
Number of flare-up episode
Assessment by CAJIS
Base
Study type
Interventional
Study design
Basic design
Parallel
Randomization
Randomized
Randomization unit
Individual
Blinding
Double blind -all involved are blinded
Control
Placebo
Stratification
YES
Dynamic allocation
YES
Institution consideration
Institution is not considered as adjustment factor.
Blocking
YES
Concealment
Central registration
Intervention
No. of arms
2
Purpose of intervention
Treatment
Type of intervention
| Medicine |
Interventions/Control_1
Initial dose is 1 mg per day for patients with body surface area less than 1.5 m2, or 2 mg per day for patients with that not less than 1.5 m2. Oral intake will be adjusted targeting trough value between 5 and 15 ng/ml. Maximum dose is 4 mg per day.
Interventions/Control_2
Placebo-control. For placebo group, oral intake will be adjusted randomly.
Interventions/Control_3
Interventions/Control_4
Interventions/Control_5
Interventions/Control_6
Interventions/Control_7
Interventions/Control_8
Interventions/Control_9
Interventions/Control_10
Eligibility
Age-lower limit
| 6 | years-old | <= |
Age-upper limit
| 59 | years-old | >= |
Gender
Male and Female
Key inclusion criteria
Patients diagnosed as "Definitive" or "Probable" according to the diagnostic criteria proposed by the research group for intractalble diseases organized by MHLW.
Key exclusion criteria
1. Patients with acute or chronic infection
2. Patients with skin sore reaching dermis
3. Patients experiencing flare-up during last 90 days
4. Past usage of mTOR inhibitors or other molecular target drugs relating mTOR pathway
5. Usage of medicine or foods which are prohibited for concurrent use of Sirolimus
6. History of allergy to Sirolimus, Sirolimus derivatives or additive substance
7. Patients with HBV, with the history of HBV or with active type-C hepatitis.
8. Severe abnormality in blood or liver function
9. Uncontrollable abnormal lipid metabolism
10. Renal failure
11. Immunodeficiency including HIV or primary immunodeficiency
12. Patients who are not able to take tablet, or who have gastrointestinal disorders possibly leading to insufficient absorption of Sirolimus
13. Patients who had surgery during 8 weeks before the registry
14. Pregnant, probably pregnant, or breast-feeding women. Patients who do not agree birth control during clinical trial.
Target sample size
20
Research contact person
Name of lead principal investigator
| 1st name | Junya |
| Middle name | |
| Last name | Toguchida |
Organization
Institute for Frontier Life and Medical Sciences, Kyoto University
Division name
Department of Regeneration Science and Engineering, Laboratory of Tissue Regeneration
Zip code
606-8507
Address
53, Shogoin-Kawahara-cho, Sakyo-ku, Kyoto
TEL
075-751-4134
togjun@infront.kyoto-u.ac.jp
Public contact
Name of contact person
| 1st name | Junya |
| Middle name | |
| Last name | Toguchida |
Organization
Institute for Frontier Life and Medical Sciences, Kyoto University
Division name
Department of Regeneration Science and Engineering, Laboratory of Tissue Regeneration
Zip code
606-8507
Address
53, Shogoin-Kawahara-cho, Sakyo-ku, Kyoto
TEL
075-751-4134
Homepage URL
togjun@infront.kyoto-u.ac.jp
Sponsor or person
Institute
Kyoto University
Institute
Department
Personal name
Funding Source
Organization
Japan Agency for Medical Research and Development
Organization
Division
Category of Funding Organization
Japanese Governmental office
Nationality of Funding Organization
Other related organizations
Co-sponsor
Nobelpharma Co., Ltd.
Name of secondary funder(s)
IRB Contact (For public release)
Organization
Kyoto University Hospital Institutional Review Board
Address
54 Kawahara-cho, Shogoin, Sakyo-ku, Kyoto, 606-8507, JAPAN
Tel
075-751-4389
tiken@kuhp.kyoto-u.ac.jp
Secondary IDs
Secondary IDs
NO
Study ID_1
Org. issuing International ID_1
Study ID_2
Org. issuing International ID_2
IND to MHLW
初回届出年月日：平成24年6月29日、届出回数：第2回
Institutions
Institutions
京都大学医学部附属病院（京都府）
東京大学医学部附属病院（東京都）
名古屋大学医学部附属病院（愛知県）
九州大学病院（福岡県）
Other administrative information
Date of disclosure of the study information
| 2017 | Year | 08 | Month | 01 | Day |
Related information
URL releasing protocol
Publication of results
Unpublished
Result
URL related to results and publications
Number of participants that the trial has enrolled
Results
Results date posted
Results Delayed
Results Delay Reason
Date of the first journal publication of results
Baseline Characteristics
Participant flow
Adverse events
Outcome measures
Plan to share IPD
IPD sharing Plan description
Progress
Recruitment status
Completed
Date of protocol fixation
| 2017 | Year | 06 | Month | 23 | Day |
Date of IRB
| 2017 | Year | 06 | Month | 30 | Day |
Anticipated trial start date
| 2017 | Year | 09 | Month | 07 | Day |
Last follow-up date
| 2021 | Year | 04 | Month | 12 | Day |
Date of closure to data entry
| 2021 | Year | 07 | Month | 26 | Day |
Date trial data considered complete
| 2021 | Year | 08 | Month | 31 | Day |
Date analysis concluded
Other
Other related information
Management information
Registered date
| 2017 | Year | 07 | Month | 28 | Day |
Last modified on
| 2023 | Year | 08 | Month | 03 | Day |
Link to view the page
Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000032495
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